Trial Outcomes & Findings for Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome (NCT NCT02629991)
NCT ID: NCT02629991
Last Updated: 2021-10-07
Results Overview
The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome.
COMPLETED
PHASE2
23 participants
Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.
2021-10-07
Participant Flow
Participant milestones
| Measure |
Intranasal Oxytocin
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
12
|
|
Overall Study
COMPLETED
|
10
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
Baseline characteristics by cohort
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.72 years
STANDARD_DEVIATION 3.5 • n=5 Participants
|
8.08 years
STANDARD_DEVIATION 3.82 • n=7 Participants
|
8.87 years
STANDARD_DEVIATION 3.68 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Baseline Height (cm)
|
141.65 cm
STANDARD_DEVIATION 15.23 • n=5 Participants
|
124.91 cm
STANDARD_DEVIATION 31.71 • n=7 Participants
|
132.92 cm
STANDARD_DEVIATION 26.11 • n=5 Participants
|
|
Baseline Weight (kg)
|
42.13 kg
STANDARD_DEVIATION 14.6 • n=5 Participants
|
38.53 kg
STANDARD_DEVIATION 20.97 • n=7 Participants
|
40.25 kg
STANDARD_DEVIATION 17.89 • n=5 Participants
|
|
Baseline BMI
|
20.61 kg/m^2
STANDARD_DEVIATION 5.04 • n=5 Participants
|
21.2 kg/m^2
STANDARD_DEVIATION 6.05 • n=7 Participants
|
20.92 kg/m^2
STANDARD_DEVIATION 5.47 • n=5 Participants
|
|
Hyperphagia Questionnaire (HQ)-Total
|
27.91 score on a scale
STANDARD_DEVIATION 10.77 • n=5 Participants
|
30.58 score on a scale
STANDARD_DEVIATION 8.08 • n=7 Participants
|
29.3 score on a scale
STANDARD_DEVIATION 9.34 • n=5 Participants
|
PRIMARY outcome
Timeframe: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Hyperphagia Questionnaire (HQ)- Total Score. The HQ is a 11-item clinician-rated instrument that was designed to measure food related behaviors in PWS. Items map onto 3 subscales; Hyperphagic Behavior, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Hyperphagia Questionnaire (HQ)- Total Score
Baseline
|
27.91 score on a scale
Standard Deviation 10.77
|
30.58 score on a scale
Standard Deviation 8.08
|
|
Hyperphagia Questionnaire (HQ)- Total Score
Week 4
|
27.4 score on a scale
Standard Deviation 9.85
|
23.33 score on a scale
Standard Deviation 8.41
|
|
Hyperphagia Questionnaire (HQ)- Total Score
Week 8
|
26.91 score on a scale
Standard Deviation 11.67
|
25.09 score on a scale
Standard Deviation 9.86
|
PRIMARY outcome
Timeframe: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Hyperphagia Questionnaire (HQ)- Behavior Factor Score assesses food seeking behaviors (e.g., stealing food). The Behavior Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviors and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Hyperphagia Questionnaire (HQ)- Behavior Factor Score
Baseline
|
9.55 score on a scale
Standard Deviation 5.01
|
9.75 score on a scale
Standard Deviation 3.47
|
|
Hyperphagia Questionnaire (HQ)- Behavior Factor Score
Week 4
|
9.4 score on a scale
Standard Deviation 4.27
|
7.22 score on a scale
Standard Deviation 2.64
|
|
Hyperphagia Questionnaire (HQ)- Behavior Factor Score
Week 8
|
9.64 score on a scale
Standard Deviation 4.9
|
8.09 score on a scale
Standard Deviation 4.25
|
PRIMARY outcome
Timeframe: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Hyperphagia Questionnaire (HQ)- Drive Factor Score assesses the persistence in asking for food. The Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Hyperphagia Questionnaire (HQ)- Drive Factor Score
Baseline
|
10.82 score on a scale
Standard Deviation 4.81
|
13.58 score on a scale
Standard Deviation 4.44
|
|
Hyperphagia Questionnaire (HQ)- Drive Factor Score
Week 4
|
11 score on a scale
Standard Deviation 4.03
|
10.44 score on a scale
Standard Deviation 5.46
|
|
Hyperphagia Questionnaire (HQ)- Drive Factor Score
Week 8
|
10.36 score on a scale
Standard Deviation 4.84
|
10.55 score on a scale
Standard Deviation 4.48
|
PRIMARY outcome
Timeframe: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported.Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Hyperphagia Questionnaire (HQ)- Severity Factor Score assesses the severity of hyperphagia. The Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Hyperphagia Questionnaire (HQ) -Severity Factor Score
Baseline
|
7.55 score on a scale
Standard Deviation 2.62
|
7.25 score on a scale
Standard Deviation 2.09
|
|
Hyperphagia Questionnaire (HQ) -Severity Factor Score
Week 4
|
7.00 score on a scale
Standard Deviation 2.54
|
5.67 score on a scale
Standard Deviation 2.45
|
|
Hyperphagia Questionnaire (HQ) -Severity Factor Score
Week 8
|
6.91 score on a scale
Standard Deviation 2.74
|
6.45 score on a scale
Standard Deviation 2.54
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score assesses ritualistic behaviors. Scores range from 0-18, with higher scores indicating more ritualistic behaviors and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
Baseline
|
6.27 score on a scale
Standard Error 3.74
|
9.42 score on a scale
Standard Error 5.00
|
|
Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
Week 4
|
4.50 score on a scale
Standard Error 3.84
|
6.27 score on a scale
Standard Error 3.77
|
|
Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score
Week 8
|
4.90 score on a scale
Standard Error 3.73
|
6.64 score on a scale
Standard Error 4.65
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score assesses the need for sameness in the environment. Scores range from 0-33, with higher scores indicating more need for sameness and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
Baseline
|
9.36 score on a scale
Standard Deviation 4.72
|
14.17 score on a scale
Standard Deviation 8.07
|
|
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
Week 4
|
6.6 score on a scale
Standard Deviation 5.25
|
8 score on a scale
Standard Deviation 6.6
|
|
Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score
Week 8
|
7.3 score on a scale
Standard Deviation 5.17
|
7.55 score on a scale
Standard Deviation 7.39
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score assesses stereotyped behaviors. Scores range from 0-18, with higher scores indicating more stereotyped behaviors and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
Baseline
|
2.18 score on a scale
Standard Deviation 2.52
|
3.33 score on a scale
Standard Deviation 2.84
|
|
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
Week 4
|
2.5 score on a scale
Standard Deviation 3.76
|
2 score on a scale
Standard Deviation 2.53
|
|
Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score
Week 8
|
2.2 score on a scale
Standard Deviation 3.01
|
1.09 score on a scale
Standard Deviation 1.58
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score assesses compulsive behaviors. Scores range from 0-24, with higher scores indicating more compulsive behaviors and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
Week 4
|
4.9 score on a scale
Standard Deviation 5.69
|
5.27 score on a scale
Standard Deviation 5.24
|
|
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
Week 8
|
4.5 score on a scale
Standard Deviation 5.99
|
5.09 score on a scale
Standard Deviation 5.41
|
|
Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score
Baseline
|
5.18 score on a scale
Standard Deviation 6.9
|
7.5 score on a scale
Standard Deviation 4.89
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score assesses self-injurious behaviors. Scores range from 0-24, with higher scores indicating more self-injurious behaviors and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
Baseline
|
5.00 score on a scale
Standard Deviation 2.97
|
2.82 score on a scale
Standard Deviation 2.86
|
|
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
Week 4
|
4.40 score on a scale
Standard Deviation 2.76
|
3.45 score on a scale
Standard Deviation 5.68
|
|
Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score
Week 8
|
4.40 score on a scale
Standard Deviation 2.46
|
3.36 score on a scale
Standard Deviation 5.78
|
SECONDARY outcome
Timeframe: Trends over multiple time points (Weeks 0, 4, and 8).Population: 1 subject discontinued oxytocin early and 1 subject discontinued placebo early
The Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score assesses restricted interests. Scores range from 0-12, with higher scores indicating more restricted interests and a worse outcome.
Outcome measures
| Measure |
Intranasal Oxytocin
n=11 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 Participants
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
Baseline
|
3.27 score on a scale
Standard Error 3.55
|
5.17 score on a scale
Standard Error 3.27
|
|
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
Week 4
|
2.20 score on a scale
Standard Error 1.81
|
4.00 score on a scale
Standard Error 3.13
|
|
Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score
Week 8
|
2.10 score on a scale
Standard Error 2.13
|
3.73 score on a scale
Standard Error 3.82
|
Adverse Events
Intranasal Oxytocin (IN-OXT)
Matched Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Intranasal Oxytocin (IN-OXT)
n=11 participants at risk
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
Matched Placebo
n=12 participants at risk
Participants were instructed to take 16 IU/per day everyday (2 puffs per nostril, 4 IU each puff).
|
|---|---|---|
|
General disorders
Fatigue
|
18.2%
2/11 • Number of events 2 • 8 Weeks
|
16.7%
2/12 • Number of events 2 • 8 Weeks
|
|
General disorders
Increased Thirst
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Renal and urinary disorders
Nocturia
|
18.2%
2/11 • Number of events 2 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Psychiatric disorders
Insomnia
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Nervous system disorders
Increased cataplexy
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Nervous system disorders
Increased movements, involuntary
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Psychiatric disorders
Irritability
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Nervous system disorders
Dizziness
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis (nose bleed)
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
General disorders
Fever
|
18.2%
2/11 • Number of events 3 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
General disorders
Scraped Knee
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
General disorders
Stomach Virus
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
General disorders
Altitute Sickness
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Infections and infestations
Shingles
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Infections and infestations
Upper Respiratory Infection
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Infections and infestations
Rhinitis Infective
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
25.0%
3/12 • Number of events 3 • 8 Weeks
|
|
Infections and infestations
Pneumonia
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Infections and infestations
Sinusitis
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
16.7%
2/12 • Number of events 2 • 8 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Infections and infestations
Otitis
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Skin and subcutaneous tissue disorders
Worsening eczema
|
9.1%
1/11 • Number of events 1 • 8 Weeks
|
0.00%
0/12 • 8 Weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/11 • 8 Weeks
|
8.3%
1/12 • Number of events 1 • 8 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place